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. Author manuscript; available in PMC: 2017 Jul 7.
Published in final edited form as: Sci Transl Med. 2016 Oct 12;8(360):360ra134. doi: 10.1126/scitranslmed.aaf9336

Figure 2. Editing of wild-type human CD34+ HSPCs by the Cas9 RNP.

Figure 2

A) Analysis of editing in un-expanded HSPCs (left) and erythroid-expanded HSPCs (right), using trG10 RNP and conditions as indicated. Templates, which are asymmetric about the G10 cut site, were designed as described in the text. All samples are 3 biological replicates, error bars are ± standard deviation. B) Modification (HDR+Indel) at off-target sites in HSPCs edited with the trG10 RNP and template T88-107, compared to untreated cells. Samples for HBB, OT1, FSCN3, and MNT are from 3 biological replicates, with error bars ± standard deviation. Targets were selected using the online CRISPR-design tool (reference). C) Indel formation at on-and off-target sites by Cas9 mutants with increased specificity (HF1 and espCas9 1.1), compared to WT Cas9, all complexed to the G10 sgRNA and no ssODN. All samples are n=3 biological replicates, with error bars ± standard deviation.